by CAP | Jun 27, 2023 | News
Amicus Therapeutics, announces the approval of Opfolda (miglustat) capsules, an enzyme stabilizer of cipaglucosidase alfa, by the European Commission for the treatment of Pompe disease.
by CAP | Jan 8, 2022 | Events
Starting Jan 16, 2022, this online series brings together Pompe disease families and other experts in five 2-hr sessions over 11 days.
by CAP | Nov 15, 2021 | News
Health Canada has approved avalglucosidase alfa (Nexviazyme™) for the long-term treatment of late-onset Pompe disease in patients over the age of 6 months.
by CAP | Aug 12, 2021 | News
In a recent announcement, the Pompe disease community learned that the U.S. Food and Drug Administration (FDA) has approved Nexviazyme® for the treatment of late-onset Pompe patients that are one year old and older.
by CAP | Feb 15, 2021 | News
Amicus Therapeutics recently announced topline results of its phase 3 PROPEL study of its combination therapy, AT-GAA, for the treatment of late onset Pompe disease (LOPD).
by CAP | Sep 20, 2020 | News, Research & Studies
An excellent review article focused on the latest developments in Pompe disease research was recently published by Naresh K. Meena and Nina Raben from the NIH.
by CAP | Jul 12, 2020 | News, Research & Studies
Results from the COMET study were recently reported showing neoGAA (avalglucosidase alfa), the newer enzyme replacement therapy (ERT) currently in development, to be as effective as the currently approved ERT, Myozyme (alglucosidase alfa), in persons with late-onset Pompe disease.
by CAP | Mar 28, 2020 | COVID-19
At this time, Sanofi Genzyme does not anticipate shortages for patients resulting from the COVID-19 situation.
by CAP | Mar 19, 2020 | COVID-19
Patients enrolled in all clinical studies for AT-GAA for Pompe disease, including the PROPEL pivotal study, will continue to receive study drug.
by Brad Crittenden | Jan 7, 2020 | News
Amicus Therapeutics today announced clinical, regulatory and manufacturing advancements for AT-GAA.